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CER-001 Therapy as a Novel Approach to Treat Genetic Orphan Diseases (TANGO)

Primary Purpose

Familial Hypoalphalipoproteinemia

Status
Terminated
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
CER-001
Placebo
Sponsored by
Cerenis Therapeutics, SA
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Familial Hypoalphalipoproteinemia focused on measuring Low HDL, Low apoA-I, ABCA-1 mutations, ApoA-I mutations

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Main Inclusion Criteria:

  • Male and female patients, aged 18 and above.
  • ApoA-I < 70 mg/dL
  • Symptomatic or asymptomatic cardiovascular disease
  • Diagnosis of genetically confirmed HDL-c deficiency due to defects in genes coding for ABCA1 and/or ApoA-1
  • Stable doses of lipid lowering therapies for at least 6 weeks prior to baseline procedures

Main Exclusion Criteria:

  • Females of childbearing potential
  • Patients with LCAT mutations
  • Patients who experienced recent cardiovascular or cerebrovascular events
  • Hypertriglyceridemia (>500 mg/dL)
  • Severe anemia (Hgb < 10 g/dL)
  • Uncontrolled diabetes (HbA1c >10%)
  • Congestive heart failure (NYHA class II or higher)
  • Contraindication for MRI scanning (e.g., implanted metal objects, claustrophobia)

Sites / Locations

  • Investigative Site
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Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

CER-001

Placebo

Arm Description

CER-001 infusion; 9 weekly infusions followed by 20 biweekly infusions

Saline infusion; 9 weekly infusions followed by 20 biweekly infusions

Outcomes

Primary Outcome Measures

Change in mmean vessel wall area (MVWA) of the carotid artery
Change from baseline to Week 24 carotid MVWA; CER-001 versus placebo; measured by 3TMRI

Secondary Outcome Measures

Change in mean vessel wall area (MVWA) of the carotid artery
Change from baseline to Week 8 carotid MVWA; CER-001 versus placebo; measured by 3TMRI
Change in mean vessel wall area (MVWA) of the carotid artery
Change from baseline to Week 48 carotid MVWA; CER-001 versus placebo; measured by 3TMRI
Change in Target to Background Ratio (TBR) of the carotid artery
Change from baseline to Week 24 in carotid TBR; CER-001 versus placebo; measured by FDG-PET

Full Information

First Posted
February 17, 2016
Last Updated
February 6, 2019
Sponsor
Cerenis Therapeutics, SA
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1. Study Identification

Unique Protocol Identification Number
NCT02697136
Brief Title
CER-001 Therapy as a Novel Approach to Treat Genetic Orphan Diseases
Acronym
TANGO
Official Title
Phase 3, Multicenter, Randomized, 48 Week, Double Blind, Parallel Group, Placebo Controlled Study to Evaluate Efficacy and Safety of CER-001 on Vessel Wall Area in Patients With Genetically Defined Familial Primary Hypoalphalipoproteinemia
Study Type
Interventional

2. Study Status

Record Verification Date
February 2019
Overall Recruitment Status
Terminated
Why Stopped
Lack of efficacy
Study Start Date
December 2015 (Actual)
Primary Completion Date
October 2018 (Actual)
Study Completion Date
December 21, 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Cerenis Therapeutics, SA

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The purpose of this study is to assess the impact of 29 intravenous infusions of CER-001 vs. placebo, given at weekly (9 infusions) and biweekly (20 infusions) intervals on carotid vessel wall area as measured by 3TMRI, when administered to patients with familial primary hypoalphalipoproteinemia with proven CVD and appropriate background lipid-lowering therapy.
Detailed Description
Subjects will be required to have acceptable baseline 3TMRI imaging of carotid arteries. Subjects meeting all eligibility criteria will be randomized to receive CER-001 or placebo (2:1 randomization scheme). Randomized subjects will return weekly for a total of 9 infusions and then biweekly for an additional 20 infusions. 3TMRI imaging of the carotid and femoral arteries will be performed at Week 8, Week 24 (primary endpoint) and Week 48. The total study duration from randomization can range from 50 to 54 weeks for patients completing the study as designed.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Familial Hypoalphalipoproteinemia
Keywords
Low HDL, Low apoA-I, ABCA-1 mutations, ApoA-I mutations

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
30 (Actual)

8. Arms, Groups, and Interventions

Arm Title
CER-001
Arm Type
Experimental
Arm Description
CER-001 infusion; 9 weekly infusions followed by 20 biweekly infusions
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Saline infusion; 9 weekly infusions followed by 20 biweekly infusions
Intervention Type
Drug
Intervention Name(s)
CER-001
Other Intervention Name(s)
CAS 138-3435-67-3
Intervention Description
Recombinant human apoA-I/phospholipid complexes
Intervention Type
Drug
Intervention Name(s)
Placebo
Other Intervention Name(s)
Normal saline
Intervention Description
0.9% Sodium Chloride Injection, USP
Primary Outcome Measure Information:
Title
Change in mmean vessel wall area (MVWA) of the carotid artery
Description
Change from baseline to Week 24 carotid MVWA; CER-001 versus placebo; measured by 3TMRI
Time Frame
Baseline to Week 24
Secondary Outcome Measure Information:
Title
Change in mean vessel wall area (MVWA) of the carotid artery
Description
Change from baseline to Week 8 carotid MVWA; CER-001 versus placebo; measured by 3TMRI
Time Frame
Baseline to Week 8
Title
Change in mean vessel wall area (MVWA) of the carotid artery
Description
Change from baseline to Week 48 carotid MVWA; CER-001 versus placebo; measured by 3TMRI
Time Frame
Baseline to Week 48
Title
Change in Target to Background Ratio (TBR) of the carotid artery
Description
Change from baseline to Week 24 in carotid TBR; CER-001 versus placebo; measured by FDG-PET
Time Frame
Baseline to Week 24
Other Pre-specified Outcome Measures:
Title
Change in femoral MVWA
Description
Assessed by 3TMRI; change from baseline; CER-001 versus placebo
Time Frame
Baseline, Weeks 8, 24 and 48

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Main Inclusion Criteria: Male and female patients, aged 18 and above. ApoA-I < 70 mg/dL Symptomatic or asymptomatic cardiovascular disease Diagnosis of genetically confirmed HDL-c deficiency due to defects in genes coding for ABCA1 and/or ApoA-1 Stable doses of lipid lowering therapies for at least 6 weeks prior to baseline procedures Main Exclusion Criteria: Females of childbearing potential Patients with LCAT mutations Patients who experienced recent cardiovascular or cerebrovascular events Hypertriglyceridemia (>500 mg/dL) Severe anemia (Hgb < 10 g/dL) Uncontrolled diabetes (HbA1c >10%) Congestive heart failure (NYHA class II or higher) Contraindication for MRI scanning (e.g., implanted metal objects, claustrophobia)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Erik SG Stroes, MD PhD
Organizational Affiliation
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Official's Role
Study Chair
Facility Information:
Facility Name
Investigative Site
City
Salt Lake City
State/Province
Utah
Country
United States
Facility Name
Investigative Site
City
La Louvière
Country
Belgium
Facility Name
Investigative Site
City
Vancouver
State/Province
British Columbia
Country
Canada
Facility Name
Investigative Site
City
Halifax
State/Province
Nova Scotia
Country
Canada
Facility Name
Investigative Site
City
London
State/Province
Ontario
Country
Canada
Facility Name
Investigative Site
City
Chicoutimi
State/Province
Quebec
Country
Canada
Facility Name
Investigative Site
City
Montreal
State/Province
Quebec
Country
Canada
Facility Name
Investigative Site
City
Lille
Country
France
Facility Name
Investigative Site
City
Montpellier
Country
France
Facility Name
Investigative Site
City
Rouen
Country
France
Facility Name
Investigative Site
City
Toulouse
Country
France
Facility Name
Investigative Site
City
Jerusalem
Country
Israel
Facility Name
Investigative Site
City
Tel Aviv
Country
Israel
Facility Name
Investigative Site
City
Genoa
Country
Italy
Facility Name
Investigative Site
City
Milan
Country
Italy
Facility Name
Investigative Site
City
Pisa
Country
Italy
Facility Name
Investigative Site
City
Rome
Country
Italy
Facility Name
Investigative Site
City
Amsterdam
Country
Netherlands
Facility Name
Investigative Site
City
Utrecht
Country
Netherlands

12. IPD Sharing Statement

Plan to Share IPD
Undecided
IPD Sharing Plan Description
To be determined

Learn more about this trial

CER-001 Therapy as a Novel Approach to Treat Genetic Orphan Diseases

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